Study of BMN 351 given by intravenous infusion to evaluate safety and tolerability in patients with Duchenne Muscular Dystrophy

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What is this study about?

This study focuses on testing a new medication called BMN 351 in patients with Duchenne Muscular Dystrophy, a genetic condition that causes progressive muscle weakness and loss of muscle tissue. The purpose of this research is to evaluate how safe the medication is and how well patients can tolerate different dose levels when given through intravenous infusion.

The study will test BMN 351, which is a special type of molecule called a phosphorothioate oligonucleotide that targets a specific part of genetic material called exon 51. The medication is given as a concentrated solution that is diluted and administered directly into the bloodstream through an intravenous line. During the study, participants will receive multiple doses of the medication, and researchers will monitor how the body processes the drug.

Throughout the study, researchers will collect information about any side effects that may occur and perform various medical tests to ensure participant safety. They will also measure how the medication moves through the body and how it affects muscle tissue. This is an open-label study, which means all participants will receive the active medication, and the dose will be gradually increased over time to find the most appropriate amount.

1 Initial assessment and screening

Confirmation of Duchenne muscular dystrophy diagnosis through genetic testing

Basic health checks including urinalysis and physical examination

Verification of ability to walk independently and complete a 10-meter walk/run test in 8 seconds or less

Confirmation of stable corticosteroid or vamorolone treatment for at least 12 weeks prior

2 Treatment initiation

Start of BMN 351 administration through intravenous infusion

The medication will be given as a concentrate that is diluted for infusion into a vein

Different dose levels will be tested to determine safety and effectiveness

3 Regular monitoring during treatment

Regular physical examinations throughout the study period

Safety laboratory tests to monitor health status

Heart monitoring through ECG (electrical heart activity recording)

Heart ultrasound (echocardiography) examinations

Blood and urine samples to measure medication levels

Muscle tissue analysis to check medication concentration

4 Ongoing medication requirements

Continuation of current corticosteroid or vamorolone treatment

Dose adjustments of current medications only if needed due to weight changes

The study will continue until approximately August 2026

5 Safety monitoring

Continuous monitoring for any side effects or health changes

Regular assessment of breathing function to ensure no ventilation support is needed

Documentation of any unexpected health events during the study period

Who Can Join the Study?

Must be a male child aged between 4 and 10 years old at the time of initial screening
Must have a confirmed diagnosis of Duchenne muscular dystrophy with a specific genetic mutation that can be treated with exon 51 skipping (this will be verified by a genetic counselor)
Must be able to walk independently without using any walking aids and complete a 10-meter walk/run test in 8 seconds or less
Must not need breathing support devices during the day and should not be expected to need them within the next year
Must be currently taking oral corticosteroids or vamorolone (medications that help manage muscle weakness) at a stable dose for at least 12 weeks before starting the study. The dose can only be adjusted for weight changes during the study
Must have normal urine test results during screening (a small amount of protein in urine is acceptable)

Who Cannot Join the Study?

History of any medical condition that would make the patient unsuitable for the study, as determined by the study doctor
Prior participation in any other clinical trial within 30 days before starting this study
Female patients (study is only open to male participants)
Age outside the children and adolescents range
Known allergies to the study medication or similar substances
Presence of other significant medical conditions that could interfere with the study results
Unable to follow study procedures or attend required study visits
Currently taking medications that could interact with the study drug
Previous treatment with gene therapy or cell therapy
Significant abnormal laboratory test results that could affect safety
Major surgery within 3 months before starting the study
Active infections or other serious illnesses
Mental conditions that could affect the ability to provide informed consent or follow study procedures

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome	Italy

Other Sites

Site Name	City	Country
Ospedale San Raffaele S.r.l.	Milan	Italy
Hospital Sant Joan De Deu Barcelona	Esplugues De Llobregat	Spain
Cscasn Cukqrzh Ncjv	Milan	Italy
Licax Ubmprsvsudhv Mzkkstz Chwibhm (biiwj	Leiden	The Netherlands

Trial status

Country	Status	Recruitment Start
Italy	Not recruiting	30.11.2023
Spain	Not recruiting	30.11.2023
The Netherlands	Not recruiting	30.11.2023

Trial locations

BMN 351 is an investigational medication administered through intravenous (into the vein) injection. It is being studied as a potential treatment for Duchenne Muscular Dystrophy (DMD). This medication is designed to help address the underlying cause of DMD, a genetic condition that causes progressive muscle weakness. The study aims to understand how safe the medication is and how well patients tolerate it when given at different dose levels.

Investigated diseases:

Duchenne muscular dystrophy

Duchenne Muscular Dystrophy – A genetic disorder that causes progressive muscle weakness and degeneration. The condition primarily affects boys and begins in early childhood, typically between ages 2 and 6. The disease is characterized by gradual weakening of muscles, starting in the legs and pelvis before affecting other parts of the body. Children with this condition often have difficulty with basic movements like walking, running, and jumping. The muscle weakness occurs because the body cannot produce a protein called dystrophin, which is essential for building and maintaining healthy muscle tissue. As the disease progresses, it affects various muscle groups throughout the body.

Trial ID:

2023-506737-30-00

Protocol code:

351-201

NCT ID:

NCT06280209

Trial Phase:

Phase I and Phase II (Integrated) – First administration to humans

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Study of BMN 351 given by intravenous infusion to evaluate safety and tolerability in patients with Duchenne Muscular Dystrophy

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?